Building a Translational Roadmap for Next-Generation CNS Gene Therapies with TfR1 CapX

• Applying TfR1 CapX to enable broad CNS delivery of novel therapeutic modalities, including the CHARM epigenetic editing platform being advanced for prion disease in collaboration with the Broad Institute
• Demonstrating the versatility of TfR1 CapX across payload modalities while maintaining efficient brain and spinal cord exposure following intravenous administration
• Developing quantitative translational frameworks that connect biodistribution, receptor engagement, and target knockdown data to clinically relevant dose selection strategies
• Navigating regulatory expectations for first-in-class receptortargeted CNS delivery platforms and establishing a repeatable pathway for future rare disease and neurodegenerative programs

Speakers:

Christopher Davis

Director, Nonclinical & Translational Research

Apertura Gene Therapy